Innovator Spotlight

Q: What are the challenges in Alzheimer’s research that the Alzheimer’s Drug Discovery Foundation (ADDF) can help address?
The biggest challenge is translating the knowledge that we have gained about Alzheimer’s biology into effective therapies to treat patients—which is why the ADDF exclusively funds research in this translational space. While Alzheimer’s represents a huge market, clinical trials for Alzheimer’s cost hundreds of millions of dollars, and there are still many unknowns, making it very high risk for the pharmaceutical industry to invest in multiple new and innovative therapies. In order to continue filling the pipeline with fresh ideas and new drugs, the ADDF seed funds these projects to bring them to a stage where they become less risky from the point of view of other investors.

Drug discovery is also a team effort, often requiring collaboration between academia, biotech, pharma, venture capital, government, and other sectors. The ADDF is ideally positioned to facilitate collaboration among these sectors, addressing unmet needs like biomarkers , issues like repurposing, and strategies to accelerate translation from preclinical into the clinic for Alzheimer’s patients. The ADDF brings not just funding, but also expertise, project management, and a network to help accelerate promising research programs through the so-called “valley of death.”The ADDF describes itself as a “biomedical venture philanthropy.

Q: Why do you focus on structuring grants as investments?
Because the ADDF provides grantees with funding, third-party validation, and networking opportunities—all elements critical to the success of a drug discovery program—we seek a return to share in their success by structuring our grants as investments. Structuring our grants in this way means that when projects are successful, we receive a return on investment that can be reinvested into new and exciting drug discovery projects. Nonetheless, we don’t make investment decisions based on profit and, given the risky nature of the investment space, we do not expect returns from most of the projects that we fund.

We also believe that treating our grants as investments creates a mindset of proactive project management, ensuring that the research we fund moves forward as efficiently and effectively as possible. We feel we owe it to our donors to “get the most bang for our buck” and invest wisely for the sole purpose of developing new Alzheimer’s treatments.

Q: ADDF ACCESS connects researchers with contract research organizations and drug discovery expertise. Why was it important for ADDF to create this resource?
The idea was born out of our researchers asking us for advice on contract research organizations (CROs) and requesting more resources on how to best work with CROs. While a lot of this information is accessible for industry researchers, there are very few resources for academic investigators entering the drug discovery and development space. ADDF ACCESS creates a centralized place where vetted CROs with expertise in the central nervous system can connect with investigators in our network. Certain CROs also provide special discounts or free consulting services to ADDF ACCESS network members.  The site also hosts extensive educational material—from quick reference documents on evaluating and working with CROs to instructive videos and Webinars on the drug development process. ADDF ACCESS is free and easy for any scientist from academia or biotech to join, and we hope it will become a widely used and helpful resource for the community. [View more about ADDF ACCESS in a Huffington Post article.]

Q: What do you consider the greatest accomplishments of ADDF?
Since 1999, the ADDF has provided more than $70 million dollars to support over 450 drug discovery, development, and early detection research programs. In an outcomes report analyzing over 10 years of ADDF funding data, we found that every dollar that the ADDF has invested has stimulated $35 more from government, pharma, and private investors to further the research. Of the $45 million that the ADDF invested prior to 2011, these projects have gone to on to raise nearly $2 billion in follow-on commitments. Thirty percent of the projects we have funded have gone on to create new intellectual property. In sum, our funding has resulted in nearly 200 patents and thousands of published papers.

These numbers represent a many exciting drug discovery and diagnostic advancements for Alzheimer’s disease. For example, from 2000 to 2003, the ADDF provided over $350,000 to researchers at the University of Pennsylvania to fund the development of an accurate diagnostic test for Alzheimer’s disease. The research project eventually led to the formation of a new company, Avid Radiopharmaceuticals, which continued to advance the diagnostic test. Avid Radiopharmaceuticals was later acquired in an $800 million deal by Eli Lilly and Company and, in 2012, the company’s brain imaging test became the first FDA-approved diagnostic test for Alzheimer’s disease. The ADDF’s early funding was critical to the development of this diagnostic test and resulted in a return on investment that has since supported additional Alzheimer’s drug discovery and development research.

Similarly, more than 10 years ago the ADDF provided funding to Dr. Frank Longo to support a virtual screening approach to identify novel compounds that could block neurodegeneration in Alzheimer’s patients. This ADDF funding helped to trigger follow-on investments from the National Institutes of Health and from private donors, ultimately enabling Longo to spin out a virtual biotechnology company to continue developing these drug candidates, one of which is now entering Phase 2 clinical trial.

Q: What are your top research goals in 2015, and what will it take to reach those goals?
In 2015, the ADDF plans to continue diversifying and expanding our research portfolio. We hope to continue building our current programmatic partnerships with organizations like the Harrington Discovery Institute and Alzheimer’s Society UK while continuing to build new partnerships. In addition, the ADDF plans to expand our efforts in prevention through our newly launched website, www.CognitiveVitality.org, and through targeted funding for Alzheimer’s prevention science.

Finally, we want to move more of our research programs closer to human clinical trial testing, where the true test of efficacy lies. We owe it to the patients and families who support us to persevere and find a treatment that slows disease progression and benefits those suffering from Alzheimer’s or at risk of developing the disease.